Every challenge. Every day. Every patient.

Explore the connections across the drug discovery and development continuum- linking patients with their life-saving drugs.


When Evie was born, she wasn't expected to survive due to the rare metabolic bone disease hypophosphotasia (HPP), which affects bone growth, breathing, and mobility. At the time of her birth, there was no treatment.

By the numbers


babies born with severe HPP


ALPL gene mutations identified in patients with HPP

Rare Disease

Between 25 and 35 million Americans have a rare disease and 50% are pediatric patients. That's why Charles River partners with nonprofits, academia, and industry to discover novel drugs and methods of study for diseases like Huntington's and muscular dystrophy. We also validate mouse models to reveal new functional and behavioral endpoints in preclinical animal models.

By the numbers


worldwide affected by rare disease

6,000 - 7,000

rare diseases are known today


Alexion is a global biopharmaceutical company whose life-changing therapies serve patients and families affected by rare diseases. Specializing in innovation, development, and commercialization, Alexion partners with Charles River for safety assessment, as well as clinical laboratory and manufacturing support.

By the numbers


transformative therapies


rare diseases


countries in a global operating platform


employees worldwide

Asfotase alfa

Asfotase alfa is an enzyme replacement therapy approved to treat patients like Evie with perinatal/infantile or juvenile-onset hypophosphotasia (HPP). It works by restoring the alkaline phosphotase (ALP) enzyme in order to promote and improve bone health.

Asfotase alfa was brought to market in October 2015 after being granted Breakthrough Therapy, Priority Review, and Orphan Drug designations by the U.S. Food and Drug Administration.

By the numbers

< 200,000

people affected for a therapy to gain Orphan Status

1st and Only

prescription medicine available for hypophosphatasia


Discovery is a critical phase in the life of any new drug. Multi-disciplinary teams of chemists, biologists, pharmacologists, and toxicologists rely on the latest technologies and platforms to advance our partners' discovery programs across all major therapeutic areas, from hit ID to IND.

By the numbers


preclinical candidates delivered since 2001

> 350

patents generated for our partners


Find out more about Evie’s story and rare disease drug development.

Featured Story:
Evie Meets the Team

Go behind the scenes as Evie meets team that developed her life-saving drug.

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Probing the Genes of Rare Disease

How gene therapy and genomics are changing the game for rare diseases: a video discussion with experts from the Charles River's World Congress on Animal Models.

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Featured Story:
Partnership with Soft Bones

Working to provide valuable information, education, and support for people living with hypophosphatasia (HPP), their families, and caregivers.

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